Immunomodulatory, liver depot gene therapy for Pompe disease

[Display omitted] •ERT has been ineffective for Pompe disease patients who form high, sustained antibody titers.•AAV-mediated liver-specific expression induced tolerance to ERT through activation of Tregs.•The liver depot of GAA is more efficient than the direct transduction of muscle. Pompe disease...

Ausführliche Beschreibung

Gespeichert in:

Bibliographische Detailangaben
Veröffentlicht in:	Cellular immunology 2019-08, Vol.342, p.103737-103737, Article 103737
Hauptverfasser:	Bond, J.E., Kishnani, P.S., Koeberl, D.D.
Format:	Artikel
Sprache:	eng
Schlagworte:	Acid alpha-glucosidase alpha-Glucosidases - genetics alpha-Glucosidases - therapeutic use Animals Antibody response Dependovirus - genetics Enzyme Replacement Therapy Gene therapy Genetic Therapy - methods Glycogen storage disease Glycogen Storage Disease Type II - therapy Humans Immune tolerance Immunomodulation Liver - metabolism Mice Pompe disease Transduction, Genetic
Online-Zugang:	Volltext
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!

Schreiben Sie den ersten Kommentar!